27th Feb 2024 A plain language summary: BI 1015550 for idiopathic pulmonary fibrosis and progressive pulmonary fibrosis
27th Feb 2024 A plain language summary looking at spesolimab treatment for the prevention of flares in people with generalized pustular psoriasis (GPP)
14th Feb 2024 Long-term benefits of enzyme replacement therapy in children and teenagers with Gaucher disease type 3- a plain language summary
22nd Jan 2024 The VANGUARD study: a plain language summary looking at garadacimab for the prevention of hereditary angioedema attacks:
22nd Jan 2024 The LAVENDER study: a plain language summary looking at trofinetide treatment for Rett syndrome
22nd Jan 2024 A plain language summary of the challenges and solutions for people with antibodies against AAV-based gene therapy
17th Jan 2024 Looking at whether genetic testing can help doctors diagnose the severity of MPS I- a plain language summary
20th Nov 2023 A plain language summary comparing pegcetacoplan with supportive care for 26 weeks in participants with paroxysmal nocturnal hemoglobinuria
16th Nov 2023 A plain language summary on the impact of itch on the quality of life in people with primary biliary cholangitis
14th Nov 2023 Plain Language Summary of Publication article: the GENEr8-1 study of valoctocogene roxaparvovec gene therapy for hemophilia A
2nd Nov 2023 A plain language summary of the intergenerational promotion of health behaviours in Portuguese families affected by familial amyloid polyneuropathy
22nd Sep 2023 A plain language summary looking at marstacimab treatment for people with severe hemophilia A or B
31st Jul 2023 Plain Language Summary: recommendations for managing diarrhea caused by trofinetide use in individuals with Rett Syndrome
25th Jul 2023 Plain Language Summary: the PEGASUS study comparing pegcetacoplan with eculizumab for 16 weeks in people with paroxysmal nocturnal hemoglobinuria
22nd May 2023 Plain Language Summary: Is ravulizumab a suitable long-term treatment option for patients with paroxysmal nocturnal hemoglobinuria?
4th Apr 2023 Plain language summary of the design of the APPEAR-C3G study which showed how iptacopan works in people with complement 3 glomerulopathy
1st Mar 2023 The effects of palovarotene in patients with fibrodysplasia ossificans progressiva: a plain language summary
31st Jan 2023 Plain language summary of a study looking at the age at diagnosis and time to start of treatment in individuals with mucopolysaccharidosis type I (MPS I)
25th Nov 2022 The impact of fibrodysplasia ossificans progressiva (FOP) on patients and their family members: results from an international burden of illness survey
25th Nov 2022 Plain language summary of a study looking at the age at diagnosis and time to start of treatment in individuals with mucopolysaccharidosis type I (MPS I)
25th Nov 2022 Plain language summary of the International Collaborative Gaucher Group Gaucher Risk Assessment for Fracture score in people living with Gaucher Disease Type 1
25th Nov 2022 Plain language summary: How the Pompe Registry is helping to identify and explain gene changes in Pompe disease
20th Sep 2022 Plain language summary of a study looking at heart muscle thickness and kidney function in women with Fabry disease who received agalsidase beta treatment
20th Jun 2022 Prednisone and deflazacort in Duchenne muscular dystrophy: a patient perspective and plain language summary publication of the Cincinnati study
20th May 2022 Plain language summary of the MPOWERED trial comparing the effectiveness, safety, and patient experiences’ of monthly injections and oral medication used to treat acromegaly
1st Dec 2021 Plain language summary of the long-term effect of ataluren in patients with Duchenne muscular dystrophy